CLINICAL TRIALS PROFILE FOR CYCLOSPORINE

505(b)(2) Clinical Trials for cyclosporine

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Combination	NCT00373815 ↗	Everolimus in Combination With Cyclosporine A and Prednisolone for the Treatment of Graft Versus Host Disease	Terminated	University Hospital Tuebingen	Phase 1	2006-09-01	The present protocol is a dose-finding and toxicity study in preparation of a randomised study comparing current standard treatment CSA/prednisolone with the new combination CSA/prednisolone/everolimus.
New Formulation	NCT02961608 ↗	Conversion Pharmacodynamic Study in Stable Renal Transplant Patients Receiving Tacrolimus Two Times a Day to a New Formulation of Tacrolimus - LCP Tacro - 1 Time a Day.	Completed	Hospital Universitari de Bellvitge	Phase 4	2016-05-01	LCP-Tacro is an extended-release formulation of tacrolimus designed for once-daily dosing. Phase 1 studies demonstrated greater bioavailability than twice-daily tacrolimus capsules and no new safety concerns. - Stable kidney transplant patients can be safely converted from Adoport® twice-daily to LCP-Tacro®. - The greater bioavailability of LCP-Tacro after once-daily dosing results in similar (AUC) exposure, at a dose approximately 30% less, than the total daily dose of Adoport®. - LCP-Tacro provides a slow drug release and this results in flatter kinetics characterized by significantly lower peak-trough fluctuations. - CN is the major cellular target of the calcineurin inhibitors (CNIs) cyclosporine A (CsA) and tacrolimus. The ability of these drugs to inhibit CN activity is dependent on their binding to the respective immunophilins, cyclophilins A and B for CsA and FKBP12 for tacrolimus. - CN inhibition is a rate limiting phenomenon. Over concentrations of tacrolimus does not correlate with an increase in the CN activity.
OTC	NCT04515329 ↗	Tear Film Markers in Dry Eye Syndrome	Not yet recruiting	Sun Pharma Global FZE	Phase 4	2021-12-01	Dry eye is the most common reason for visit to an ophthalmologist's office. The prevalence is on the rise and is mainly attributed to factors such as increased environmental pollution and contact lens use. The current management options are limited to over the counter artificial tear drops and three FDA-approved drugs. Of these, cyclosporine has been used worldwide for treating mild to moderate dry eyes. The earlier version consisted of 0.05% cyclosporine which worked well for a limited number of inflammatory dry eye conditions. Recently, 0.09% cyclosporine was approved by the FDA. The nearly double concentration is expected to be more beneficial for severe inflammation which is often seen in Sjögren syndrome and other Rheumatological conditions associated with dry eyes. In this pilot project, the investigator proposes to evaluate the change in expression of SLURP1 and other markers of ocular surface inflammation before and after treatment with 0.09% cyclosporine eye drops.
OTC	NCT04515329 ↗	Tear Film Markers in Dry Eye Syndrome	Not yet recruiting	Vishal Jhanji	Phase 4	2021-12-01	Dry eye is the most common reason for visit to an ophthalmologist's office. The prevalence is on the rise and is mainly attributed to factors such as increased environmental pollution and contact lens use. The current management options are limited to over the counter artificial tear drops and three FDA-approved drugs. Of these, cyclosporine has been used worldwide for treating mild to moderate dry eyes. The earlier version consisted of 0.05% cyclosporine which worked well for a limited number of inflammatory dry eye conditions. Recently, 0.09% cyclosporine was approved by the FDA. The nearly double concentration is expected to be more beneficial for severe inflammation which is often seen in Sjögren syndrome and other Rheumatological conditions associated with dry eyes. In this pilot project, the investigator proposes to evaluate the change in expression of SLURP1 and other markers of ocular surface inflammation before and after treatment with 0.09% cyclosporine eye drops.

All Clinical Trials for cyclosporine

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000524 ↗	Myocarditis Treatment Trial	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	1986-07-01	To determine whether immunosuppressive treatment improved cardiac function in patients with biopsy-proven myocarditis.
NCT00000524 ↗	Myocarditis Treatment Trial	Completed	University of Utah	Phase 2	1986-07-01	To determine whether immunosuppressive treatment improved cardiac function in patients with biopsy-proven myocarditis.
NCT00000880 ↗	A Study to Test the Effect of Cyclosporine on the Immune System of Patients With Early HIV Disease	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	The purpose of this study is to determine the safety and effectiveness of low doses of cyclosporine (CsA) in patients with early HIV infection and to evaluate its effect on the immune system. Activation of T cells (cells of the immune system) leads to HIV replication. Inhibition of immune activation is therefore a potentially important area of therapy for patients with early HIV infection. CsA is capable of decreasing T cell activation, which in turn may decrease HIV replication.
NCT00000936 ↗	A Study To Test An Anti-Rejection Therapy After Kidney Transplantation	Terminated	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	1999-11-01	Kidney transplantation is often successful. However, despite aggressive anti-rejection drug therapy, some patients will reject their new kidney. This study is designed to test two anti-rejection approaches. Two medications in this study are currently used in children, but there is no information regarding which drug is safer or more effective. Survival rates in renal transplantation are unacceptably low. Therefore, there is a need for an improved post-transplant treatment, such as the induction therapy used in this study.

Clinical Trial Conditions for cyclosporine

Condition Name

Condition Name for cyclosporine
Intervention	Trials
Leukemia	108
Myelodysplastic Syndromes	74
Lymphoma	68
Kidney Transplantation	60

Condition MeSH

Condition MeSH for cyclosporine
Intervention	Trials
Leukemia	175
Syndrome	144
Myelodysplastic Syndromes	143
Preleukemia	138

Clinical Trial Locations for cyclosporine

Trials by Country

Trials by Country for cyclosporine
Location	Trials
Canada	163
Italy	143
China	120
Germany	94
France	82

Trials by US State

Trials by US State for cyclosporine
Location	Trials
California	138
Washington	115
Maryland	100
New York	97
Texas	89

Clinical Trial Progress for cyclosporine

Clinical Trial Phase

Clinical Trial Phase for cyclosporine
Clinical Trial Phase	Trials
PHASE4	16
PHASE3	4
PHASE2	19

Clinical Trial Status

Clinical Trial Status for cyclosporine
Clinical Trial Phase	Trials
Completed	649
RECRUITING	125
Terminated	102

Clinical Trial Sponsors for cyclosporine

Sponsor Name

Sponsor Name for cyclosporine
Sponsor	Trials
National Cancer Institute (NCI)	175
Fred Hutchinson Cancer Research Center	82
National Heart, Lung, and Blood Institute (NHLBI)	63

Sponsor Type

Sponsor Type for cyclosporine
Sponsor	Trials
Other	1186
Industry	418
NIH	293

Clinical Trials Update and Market Projection: Cyclosporine

Last updated: April 25, 2026

Cyclosporine is a first-in-class calcineurin inhibitor with a long-established global market footprint driven by transplantation immunosuppression and a steady base in autoimmune indications that still use cyclosporine formulations (notably topical for ocular and systemic for selected autoimmune diseases). The clinical pipeline is concentrated in next-generation delivery and indication refinements rather than new cyclosporine molecular entities.

Where are cyclosporine trials today (what is actually moving)?

Clinical trial activity map by segment

Clinical development for cyclosporine is dominated by formulation and regimen studies across these segments:

• Transplantation (core): peri-transplant induction/maintenance regimen comparisons, TDM-driven dosing studies, and conversion studies between formulations to improve exposure consistency.
• Ophthalmology (core for branded topical): cyclosporine ophthalmic solutions/emulsions and comparative safety/tolerability trials targeting dry eye disease and ocular inflammation endpoints.
• Dermatology/autoimmune (variable by country): topical or systemic cyclosporine studies, often focusing on switching strategies, dose optimization, and safety monitoring.

Regimen and exposure-control themes

Across cyclosporine trial designs, the recurring motifs are:

• Therapeutic drug monitoring (TDM) alignment: trials compare outcomes across dosing strategies and monitoring practices.
• Switching and conversion: studies evaluate bioequivalence, exposure stability, and immunologic outcomes when patients switch between cyclosporine product types.
• Safety endpoint refinement: renal function, blood pressure, lipid changes, and infection rates remain central due to the known risk profile.

What the trial record implies commercially

The trial pattern indicates cyclosporine’s life-cycle strategy is “keep and optimize” rather than “replace the molecule.” That limits upside from radical efficacy gains and concentrates value in:

• maintaining guideline relevance,
• reducing formulation variability,
• and retaining reimbursement through safety and exposure consistency.

How big is cyclosporine’s market today, and what segments matter most?

Market drivers

Cyclosporine market demand is primarily shaped by:

1. Transplant volumes

   • Solid organ transplantation is the durable base.
   • Ongoing transplant activity in kidney, liver, heart, and lung maintains cyclosporine as a standard component.

2. Ocular inflammation and dry eye demand

   • Topical cyclosporine remains a frequent choice in chronic ocular surface inflammation.
   • Demand is influenced by the prevalence of dry eye disease and long-term adherence.

3. Chronic autoimmune use

   • Systemic cyclosporine is used for selected autoimmune indications, but share is sensitive to competition from newer immunomodulators and biologics.

Competitive landscape

Cyclosporine competes along three axes:

• Innovator formulations vs generics (systemic cyclosporine and topical products)
• Other calcineurin inhibitors
• Non-calcineurin immunosuppressants in transplant and targeted immunotherapies in autoimmune disease

Given cyclosporine’s patent history and wide availability, much of the market is structurally price-sensitive, with differentiation anchored to:

• formulation characteristics,
• dosing convenience,
• and safety outcomes tied to exposure control.

What is the market projection for cyclosporine (2026-2035)?

Projection structure

A defensible projection for cyclosporine must be modeled by segment because the molecule’s growth rate varies by indication and formulation category.

Segment-level projection logic

• Transplantation: low-to-moderate growth, driven by transplant volume growth and patient retention.
• Ophthalmology (topical cyclosporine): moderate growth potential tied to chronic disease prevalence and expanding treatment patterns in dry eye and ocular surface inflammation.
• Autoimmune (systemic): slower growth and potential share pressure from newer therapies.

Base-case market view (directional)

• Cyclosporine market value is expected to grow at a low single-digit to low-to-mid single-digit CAGR over the next decade, with total demand supported by transplantation and ocular chronic use.
• Value growth depends more on mix and pricing (formulation shifts, branded-to-generic dynamics, tender outcomes) than on molecular innovation.

Key downside/upside levers

Downside

• higher use of alternative immunosuppressive regimens in transplantation
• continued generics price compression
• safety-driven restriction in specific patient subgroups

Upside

• improved tolerability and exposure consistency from optimized cyclosporine formulations
• stronger adherence and persistence in chronic ocular indications
• guideline reinforcement in settings where calcineurin inhibition is still first-line or standard-of-care

Which cyclosporine formulations drive value most (and why)?

Value hierarchy

1. Systemic transplant formulations

   • Largest absolute demand base.
   • Value tied to continued standard use and reimbursement.

2. Topical ophthalmic formulations

   • Lower absolute volume than systemic but with stable chronic consumption.
   • Value supported by long-duration treatment behavior in dry eye.

3. Other topical/systemic autoimmune uses

   • Smaller, less predictable demand.
   • Often more exposed to evolving standards of care.

Why formulation matters

Cyclosporine has a relatively narrow therapeutic window in practice, and exposure consistency is operationally important. Market share and payer coverage track to:

• predictable bioavailability,
• stable dosing,
• and documented switching outcomes.

What does the IP landscape imply for future competition?

Cyclosporine is off-patent for most major systemic molecules in key jurisdictions, which structurally limits patent-protected value growth. The competitive battleground has shifted to:

• formulation-specific intellectual property (where still present),
• clinical data packages that support specific dosing or switching strategies,
• and market access strategy.

This drives a “data-led” commercial environment where companies win by showing comparable outcomes under real-world dosing or conversion protocols rather than by building novel molecular IP.

Business implications: how to underwrite cyclosporine exposure (2016-2026 learnings applied to 2026-2035)

For R&D strategy

• If developing new cyclosporine products, the fastest path to differentiation is exposure consistency and safety comparability, not novel efficacy claims.
• Trial endpoints should prioritize renal safety, infection profile, and regimen adherence, aligned with how clinicians use cyclosporine.

For investment strategy

• Expect market growth to be steady but not explosive.
• Returns will track to execution in formulation and distribution, and to reimbursement outcomes, more than to breakthrough clinical superiority.

For BD and licensing

• Look for opportunities in lifecycle management: line extensions, switching programs, and indication-specific evidence to defend formulary position.
• Any “new entrant” cyclosporine value will likely be won on differentiated dosing convenience and payer-friendly clinical evidence rather than on new mechanism.

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Key Takeaways

• Cyclosporine clinical activity is concentrated in formulation, regimen, and switching/exposure optimization, with central endpoints tied to safety and TDM alignment.
• The market is anchored by transplant immunosuppression and supported by chronic ophthalmology demand; autoimmune systemic growth is slower and more exposed to competitive displacement.
• Market projection is low single-digit to low-to-mid single-digit CAGR directional growth driven by patient persistence, transplant volume trends, and mix shifts, with ongoing generics and alternative regimens constraining upside.

FAQs

1) What types of cyclosporine trials are most common right now?
Formulation comparison studies, switching/conversion trials, and TDM-aligned regimen studies, with primary endpoints focused on safety, exposure consistency, and regimen tolerability.

2) Which cyclosporine segment is most stable for demand?
Transplantation, because ongoing solid organ procedures provide a durable baseline and cyclosporine remains a standard-of-care option in many regimens.

3) Why does exposure consistency matter for cyclosporine commercially?
Cyclosporine’s real-world dosing effectiveness and safety correlate with exposure variability. Payers and clinicians prefer evidence that supports predictable outcomes when patients switch or convert between formulations.

4) What is the biggest risk to cyclosporine growth?
Price compression from generics and substitution by alternative immunosuppressants and newer therapies that can reduce cyclosporine share in specific indications.

5) Where could cyclosporine see better-than-average growth?
Ophthalmic cyclosporine in chronic ocular inflammation settings where long-term use supports persistence and where formulation improvements can reduce tolerability barriers.

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References

[1] ClinicalTrials.gov. Cyclosporine studies search results. (accessed 2026-04-25). https://clinicaltrials.gov/
[2] Drugs@FDA, FDA labeling and approvals for cyclosporine products. (accessed 2026-04-25). https://www.accessdata.fda.gov/scripts/cder/daf/
[3] EMA EPARs and product information for cyclosporine formulations. (accessed 2026-04-25). https://www.ema.europa.eu/en/medicines